European Commission Approves Hemlibra for Moderate Haemophilia A Patients

Strategic Insights -

The European Commission has approved the expansion of the Hemlibra (emicizumab) marketing authorisation in the European Union (EU) for the routine prophylaxis of bleeding episodes in people with haemophilia A without factor VIII inhibitors. The label will now include individuals with moderate disease (FVIII ëÑ1% and ëñ 5%) and a severe bleeding phenotype. Haemophilia A affects approximately 900,000 people worldwide, with around 14% having a moderate form of the disease. Approximately 85% of people with moderate haemophilia A experience bleeds within a given year, and one in three has long-term joint problems.

Key Takeaways:

  • Haemophilia A affects around 900,000 people worldwide, with approximately 14% having a moderate form of the disease.
  • Approximately 85% of people with moderate haemophilia A experience bleeds within a given year, and one in three has long-term joint problems.
  • Hemlibra is approved for prophylactic treatment in more than 110 countries for people with haemophilia A with factor VIII inhibitors.
  • Hemlibra has been studied in one of the largest clinical trial programmes for people with haemophilia A with and without factor VIII inhibitors.
  • The approval is based on the results of the phase III HAVEN 6 trial, which demonstrated effective bleed control and a favourable safety profile in people with non-severe haemophilia A without factor VIII inhibitors.
  • Approximately 15% of people with moderate haemophilia A live a bleed-free life.
  • The treatment and care of people with severe haemophilia A are well-established, but there is less information and guidance on prophylaxis for moderate haemophilia A.
  • The severity of haemophilia A is not always reflective of bleeding behaviour, and many individuals with moderate haemophilia A may not be receiving prophylactic treatments.

Statistics:

  • 900,000 people worldwide are affected by haemophilia A.
  • 14% of people with haemophilia A have a moderate form of the disease.
  • 85% of people with moderate haemophilia A experience bleeds within a given year.
  • One in three people with moderate haemophilia A has long-term joint problems.
  • 15% of people with moderate haemophilia A live a bleed-free life.
  • Hemlibra is approved for prophylactic treatment in more than 110 countries for people with haemophilia A with factor VIII inhibitors.
  • Hemlibra has been studied in one of the largest clinical trial programmes for people with haemophilia A with and without factor VIII inhibitors.

Sources:

  • Roche, "Hemlibra, already approved for severe haemophilia A in the EU, will now also provide an effective and convenient prophylactic treatment option for people with moderate haemophilia A"
  • Roche, "Hemlibra has been studied in one of the largest clinical trial programmes for people with haemophilia A with and without factor VIII inhibitors"
  • World Federation of Hemophilia, "Report on the annual global survey 2020"
  • International Society on Thrombosis and Haemostasis, "Results from the Primary Analysis of the HAVEN 6 Study"
  • European Commission, "Hemlibra, INN-emicizumab sMPC"
  • World Federation of Hemophilia, "WFH Guidelines for the Management of Hemophilia, 3rd edition"
  • American Medical Association, "Haemophilia A"
  • National Library of Medicine, "Haemophilia A"